A New Wave of Innovation in Neovascular Eye Disease

About PanOptica

PanOptica is working to overcome the limitations of current treatments for serious ophthalmic diseases by developing a topical eye drop alternative to intravitreal (back-of-the eye) injection therapies

About PAN-90806

We are preparing to begin the next phase of clinical research for our most advanced therapy, PAN-90806

About PanOptica

Driven to help patients see their very best throughout their lives

PanOptica, Inc., is a private, clinical-stage biopharmaceutical company focused on developing a topical eye drop for the treatment of sight-threatening eye diseases caused by abnormal or leaky blood vessels. These neovascular eye diseases include neovascular age-related macular degeneration (wet AMD), the leading cause of blindness in the Western world, and diabetic retinopathy.

Currently available treatments are effective at slowing vision loss, and may improve vision for some patients, but require careful follow up and frequent, chronic intraocular injections for many patients in order to optimize vision outcomes. Patients who stop treatment may suffer debilitating vision loss.

At PanOptica, we understand the critical role vision plays in a patient’s quality of life, and we know the frustration that ophthalmologists face when they are unable to treat their patients successfully, and for patients and families for whom the burden of chronic treatment can be overwhelming.

We are driven to develop new ocular therapies that help patients see their very best throughout their lives. Our management team and board of directors bring extensive experience discovering, evaluating, developing, and commercializing ophthalmic drugs. These novel therapies may include improvements to existing medicines or entirely new treatments for previously untreatable disorders.

About PAN-90806

PAN-90806 is a novel, topically applied, once daily eye drop for the treatment of wet AMD and other neovascular eye diseases. PAN-90806 is a small molecule that blocks activation of the vascular endothelial growth factor (VEGF) receptor 2 through inhibition of the receptor’s tyrosine kinase activity. VEGF is a protein that plays a critical role in angiogenesis (the formation of new blood vessels) and increased permeability (leakage from blood vessels), two pathological processes that contribute to the vision loss associated with wet AMD.

PAN-90806 is absorbed in the blood vessels at the periphery of the eye and rapidly delivered around the eye – not through it – to the central choroid and central retina where the abnormal overproduction of VEGF does its damage in wet AMD and diabetic retinopathy.

PAN-90806’s special and specific physical chemistry plays a major role in its ability to deliver high and sustained concentration in these target tissues that enable effective once-daily dosing in order to block the damaging effects of pathological VEGF in these diseases.

In the first Phase 1/2 clinical trial of PAN-90806, data suggested signals of positive biological response to topical PAN-90806 in approximately 45-50% of 20 patients treated for up to 8 weeks with 2 different doses including improvement of vascular leakage, lesion morphology and vision.

As a result of encouraging early data from this small study, PanOptica has developed a new formulation of PAN-90806 which has shown the potential to improve comfort and tolerability for patients and improve the potential to safely deliver effective concentrations of the active ingredient to the target tissues in the choroid and retina based on preclinical data.

PanOptica plans to enter a new Phase 1/2 clinical trial for the new formulation of PAN-90806 in late 2017 or early 2018.

In addition to the clinical data from the previous Phase 1/2 clinical trial, a broad range of preclinical research supports the potential role of PAN-90806 for the treatment of neovascular eye diseases. Key evidence includes:

Pharmacokinetic studies showed topical administration of PAN-90806 achieved significant and sustained levels in the retina and choroid of multiple species after topical administration

Extensive animal pharmacology, toxicology and formulation development further characterized the profile of PAN-90806

Current Limitations in Treatment Options

PanOptica seeks to use eye drop to overcome current limitations

What are the current limitations of treatment options for wet AMD?

The pharmaceutical industry’s significant investment in ophthalmic diseases is relatively recent. The first anti-VEGF pharmacotherapy for wet AMD, the leading cause of blindness worldwide, was introduced less than 15 years ago. PanOptica and other researchers are now working to apply advances in other therapeutic categories to ophthalmology by repurposing therapies developed for diseases ranging from cancer to inflammation.

Drug delivery is a key challenge in developing effective, safe, and convenient therapies for eye diseases. Nearly 100 years ago, the first therapy for glaucoma, a common ocular disorder, was made widely available. This treatment came in the form of an eye drop, which remains the most popular delivery mechanism for ophthalmic medicine today because it is easy to administer and comfortable for the patient. Unfortunately, some chemical compounds do not lend themselves well to eye drop delivery. Some are highly water insoluble, while other molecules are too large or unstable and can degrade quickly at room temperature.

Today, newer technologies are becoming available that may overcome this challenge. Some of these technologies hold the promise of extended-release delivery, potentially improving efficiency, and reducing patient burden. Other technologies may help deliver medication internally to the eye, potentially enhancing surgical outcomes. Our objective is to develop a topical eye drop for the treatment of wet AMD and other neovascular eye diseases, including diabetic retinopathy.

Injected anti-VEGF biologics have revolutionized treatment of wet AMD and diabetic. However, the currently approved standards medicines used in treating AMD rely on fixed injection regimens in order to optimize vision outcomes.

PAN-90806: A Novel Approach to Treating Serious Eye Diseases

What differentiates PAN-90806 from current treatment options?

PAN-90806 may offer potential advantages over available therapies because it is a once daily eye drop for the treatment of wet AMD and other neovascular eye diseases, for which there are no approved eye drop treatment options. Importantly, it blocks VEGF signaling, which has been shown to be the only proven mechanism for the treatment of these serious eye diseases. However, our method is different than current treatment options. We block the effect of VEGF at the receptor and do not bind VEGF itself.

There is a significant unmet need for patients with these serious eye diseases, and PAN-90806 has the potential to reduce both the physical and emotional burden of this disease for both patients and caregivers to improve patients’ quality of life. The benefits include:

Accessible anywhere, any time (self-administered daily dose, does not require injections)

Empowers patients and caregivers

Reduces burden on caregivers

Applicable for all VEGF-treated patients who are candidates for an anti-VEGF treatments

Clinical Trials for PAN-90806

PanOptica prepares for an important new clinical trial

PanOptica is developing additional studies in wet AMD and other neovascular eye diseases, including diabetic retinopathy, utilizing a new and improved formulation. We plan to initiate the clinical trial near the end of 2017 or in early 2018, and we expect data by 1H 2019.

Management Team and Board of Directors

Paul Chaney is president and CEO of PanOptica and has nearly 30 years of experience in the pharmaceutical industry. He was responsible for Xalatan, the most widely prescribed treatment for elevated intraocular pressure (glaucoma) and the first pharmacotherapy for neovascular AMD.

Prior to co-founding PanOptica, Paul was executive vice president of OSI Pharmaceuticals and president of (OSI) Eyetech, OSI’s eyecare subsidiary. He joined Eyetech Pharmaceuticals as chief operating officer in 2003.

Paul held a variety of senior management positions at Pharmacia Corporation, including vice president of the Global Ophthalmology Business and vice president of Global Pharmaceutical Ophthalmology. He began his career as a sales representative for The Upjohn Company in 1980. Paul earned a dual degree in Biological Sciences and English from the University of Delaware.

Martin Wax, M.D.CHIEF MEDICAL OFFICER AND EXECUTIVE VICE-PRESIDENT OF RESEARCH AND DEVELOPMENT

Martin B. Wax, M.D., is chief medical officer and executive vice president of research and development at PanOptica, and a Clinical Professor of Ophthalmology at the NYU Langone Medical Center in New York City. He previously served as vice president of R&D and head of discovery research and preclinical sciences at Alcon Laboratories, where he was responsible for the creation and early development of the Alcon pharmaceutical portfolio. Dr. Wax received his medical degree from the University of Southern California in Los Angeles. He completed his residency in Ophthalmology at Wills Eye Hospital in Pennsylvania, and post-doctoral fellowships in molecular pharmacology and glaucoma at the University of Pennsylvania School of Medicine. He currently holds an appointment as a Professor of Ophthalmology and Visual Sciences at the University of Texas Southwestern Medical School in Dallas, and has been selected as one of the “Best Doctors in America” by his peers. An internationally recognized scientific expert in the field of glaucoma, Dr. Wax has co-authored more than 200 publications and serves on several scientific advisory committees and editorial boards.

Lori Forrest is the executive director of finance and controller at PanOptica. She has over 20 years of accounting experience in the pharmaceutical and consumer products industries. Her responsibilities have included financial reporting (including SEC reporting requirements and compliance with the Sarbanes-Oxley Act of 2002), budgets and forecasts, and human resource and information technology functions. Most recently, Lori was the Controller at Helsinn Therapeutics, Inc. (previously Sapphire Therapeutics, Inc.), where she played a key role in the due-diligence, merger and post-acquisition integration activities when Sapphire was purchased in January 2009. From 1997-2005, Lori worked at Vivus, Inc. in roles of increasing responsibility in both operations and corporate finance. She began her career at Carter-Wallace, Inc. after graduating from Cook College, Rutgers University with a Bachelor of Science degree in Business Economics. Lori is a certified public accountant.

David P. Bingaman, DVM, Ph.D.SENIOR DIRECTOR OF CLINICAL DEVELOPMENT

As senior director of clinical development at PanOptica, David Bingham, DVM, Ph.D., is responsible for managing PanOptica’s Retina programs, including PAN-90806, the company’s lead therapeutic candidate. David previously spent 13 years at Alcon Laboratories in Ft. Worth, Texas, where he established Ocular Angiogenesis and Diabetic Retinopathy programs, directed various aspects within Retina Development, and received the Alcon Technical Excellence Award in 2006. Dr. Bingaman earned his veterinary medical degree from Washington State University and completed an internship and residency in veterinary ophthalmology at Purdue University. He conducted his doctoral thesis in pathologic ocular angiogenesis and diabetic retinopathy in a combined program at Purdue University/Indiana University. Dr. Bingaman is a board-certified veterinary ophthalmologist and Diplomate of the American College of Veterinary Ophthalmologists.

Kristine CurtissEXECUTIVE DIRECTOR OF CLINICAL OPERATIONS

As executive director of clinical operations for PanOptica, Kristine Curtiss brings 20 years of experience in the pharmaceutical, biotechnology, and medical device industries. Previously, Kristine managed all phases of global clinical trials on strategic programs in neurology and oncology at Hoffman La-Roche, Inc. Her work there led to the approval of Tasmar® (tolcapone) and Xeloda® (capecitabine) in the United States and in Europe. Kristine also served as executive director of clinical research at Eyetech Pharmaceuticals, where she was responsible for implementing and directing the clinical program leading to the U.S. and European approvals of Macugen® (pegaptanib sodium) for neovascular age-related macular degeneration. Previously, she was director of global ophthalmology programs at Opko Health and Oraya Therapeutics, Inc. Kristine holds a Bachelor of Science degree in Chemistry from the University of Delaware.

Angela C. Kothe, O.D., PhDCLINICAL AND REGULATORY AFFAIRS CONSULTANT

Angela C. Kothe is Vice President of Silver Pharma Consulting, Inc., a firm providing clinical development, medical safety, and regulatory consulting expertise to a variety of domestic and international ophthalmology clients. Angela brings 25 years of experience in the pharmaceutical, biotechnology and medical device industry. Prior to consulting, Angela spent 19 years at Alcon Laboratories, Inc. She began her pharmaceutical career in 1993 managing clinical trials at Alcon, and in 2000, transitioned to regulatory affairs at Alcon. Her leadership in regulatory affairs led to the approval of key anti-infective, anti-inflammatory, ocular allergy and glaucoma products in the US and internationally. During the past five years, she has provided regulatory expertise to ophthalmic companies in the areas of ocular allergy, glaucoma, inflammation, retina, and dry eye. She has led several companies through the IND and NDA process, provided regulatory strategies and managed operational activities, and led meetings with the FDA and international health agencies.
Angela holds a Bachelor of Science (Life Sciences) from Queen’s University, Kingston, Ontario, Canada, and a Doctor of Optometry and PhD in visual electrophysiology from the University of Waterloo, Ontario, Canada.

David GuyerMEMBER, BOARD OF DIRECTORS

David Guyer, M.D., joined SV Life Sciences in 2006 and serves on the boards of portfolio companies Link Medicine, Lux Biosciences, NeoVista, Neurotech and Ophthotech as well as the public company Optos. David co-founded and served as CEO and director of Eyetech Pharmaceuticals Inc. for six years. He led Eyetech – a biopharmaceutical company that specialized in the development and commercialization of novel therapeutics to treat diseases of the eye – through private financing rounds totaling approximately $170 million, a $157 million initial public offering and a secondary financing. The company developed and gained FDA approval for Macugen, the first anti-VEGF treatment approved for macular degeneration. Eyetech partnered with Pfizer in a deal valued at over $750 million. OSI Pharmaceuticals subsequently acquired Eyetech in a deal valued at $935 million. Prior to founding Eyetech, Dr. Guyer was professor and chairman of the department of ophthalmology at the NYU School of Medicine. Dr. Guyer holds a bachelor of science from Yale College and a medical degree from Johns Hopkins Medical School.

Bruce PeacockMEMBER, BOARD OF DIRECTORS

Bruce Peacock has served as a member of our board of directors since March 2015. Mr. Peacock served as Chief Financial and Business Officer of Ophthotech Corporation from August 2013 to September 2014 and served as its Chief Business Officer from September 2010 to August 2013.

From April 2008 to February 2011, Mr. Peacock served as President, Chief Executive Officer and co-Chairman of the board of directors of Alba Therapeutics where he continues to serve as co-Chairman of the board of directors. Additionally, Mr. Peacock has served as a venture partner of SV Life Sciences Advisors, LLC since 2006. Previously Mr. Peacock served as the Chief Executive Officer and Director of The Little Clinic; President, Chief Executive Officer and Director of Adolor Corp. (NASDAQ: ADLR) (acquired by Cubist Pharmaceuticals, Inc. (NASDAQ: CBST)); President, Chief Executive Officer and Director of Orthovita, Inc. (acquired by Stryker Corporation); Executive Vice President, Chief Operating Officer and Director of Cephalon, Inc. (acquired by Teva Pharmaceutical Industries Ltd.); and as Chief Financial Officer of Centocor, Inc. (acquired by Johnson & Johnson (NYSE: JNJ).

Mike RossMEMBER, BOARD OF DIRECTORS

Mike Ross’ career spans over 15 years in venture capital, which followed 21 years in senior operating executive roles for leading biotechnology companies. He joined SV Health Investors as a Venture Partner in 2001 and became a Managing Partner in 2002.

Mike was the tenth employee at Genentech, where he worked for 13 years. He served as Genentech team leader for Humulin® (human insulin-Lilly), Roferon® (Interferon alpha -Roche), Protropin® (hGH), Vice President of Development during the development of Activase®, Nutropin® and Pulmozyme®. He then started Genentech’s protein engineering and small molecule discovery effort as Vice President of Medicinal and Biomolecular Chemistry. Mike was the Founding CEO of Arris Pharmaceutical, MetaXen, ExSAR and CyThera (now Viacyte). He was also Managing Partner in Didyma, LLC, a biotechnology management consulting firm. Mike served on the boards of Carta Proteomics, Epimmune, Genencor, MetaXen, and Xenova.

Mike holds an AB from Dartmouth College, a PhD in Chemistry from Caltech, and a Post-Doctoral Fellowship in Molecular Biology from Harvard.

Kevin StarrMEMBER, BOARD OF DIRECTORS

Kevin Starr is a proven operational leader with over 25 years’ experience building and operating leading biotech companies. Mr. Starr co-founded Third Rock Ventures in 2007 and focuses on the formation, development and business strategy of its portfolio companies as well as actively identifying and evaluating new investments. He also assumes active leadership roles in Third Rock portfolio companies. Prior to Third Rock Ventures, Mr. Starr was chief operating officer and chief financial officer at Millennium Pharmaceuticals, where he led efforts to raise billions of dollars in capital through strategic alliances and public financing and drove growth through company expansion and mergers and acquisitions. Earlier in his career, he served in various executive roles at Millennium BioTherapeutics, Biogen and Digital Equipment Corporation. Mr. Starr holds a master of science in corporate finance from Boston College and a bachelor of arts in mathematics and business from Colby College. He is a board member of Alnylam Pharmaceuticals and also serves on the board of directors for Afferent Pharmaceuticals, Agios Pharmaceuticals and Zafgen, Inc.

Paul ChaneyMEMBER, BOARD OF DIRECTORS

Paul Chaney is president and CEO of PanOptica and has nearly 30 years of experience in the pharmaceutical industry. He led the US and later global team that were responsible for Xalatan, the most widely prescribed treatment for elevated intraocular pressure (glaucoma) and the first pharmacotherapy for neovascular AMD.

Prior to co-founding PanOptica, Paul was executive vice president of OSI Pharmaceuticals and president of (OSI) Eyetech, OSI’s eyecare subsidiary. He joined Eyetech Pharmaceuticals as chief operating officer in 2003, where he led the team that introduced the first injectable anti-VEGF biologic treatment for treatment of wet-AMD.

Paul held a variety of senior management positions at Pharmacia Corporation, including vice president of the Global Ophthalmology Business and vice president of Global Pharmaceutical Ophthalmology. He began his career as a sales representative for The Upjohn Company in 1980. Paul earned a dual degree in Biological Sciences and English from the University of Delaware.